Project Details
Description
An abundance of genetic, experimental, and clinical evidence has implicated reduced activity of the enzyme glucocerebrosidase (GCase) in the onset of Parkinson’s disease (PD). As a result, increasing enzymatic activity of GCase is considered a promising therapeutic strategy to treat patients with PD. The goal of this collaboration is to identify novel modifiers that play a role in regulating GCase activity within the cell. These modifiers will improve our understanding of PD pathogenesis by identifying proteins and pathways involved in regulating the activity of lysosomal enzymes. Identification of druggable pathways involved in regulating GCase activity could reveal attractive new therapeutic targets for disease modification in PD. While Aim 1a (see below) is a higher priority, we feel that Aim 1b could yield valuable and likely complimentary information regarding physiological differences in dopaminergic neurons from affected and unaffected family members.
Status | Finished |
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Effective start/end date | 12/20/18 → 12/19/21 |
Funding
- AbbVie Inc. (Agmt 12/20/18)
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